| Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type B | HST32 | | |
| Burosumab for treating X-linked hypophosphataemia in children and young people | HST8 | | |
| Setmelanotide for treating obesity and hyperphagia in Bardet-Biedl syndrome | HST31 | | |
| Sebelipase alfa for treating Wolman disease | HST30 | | |
| Velmanase alfa for treating alpha-mannosidosis | HST29 | | |
| Birch bark extract for treating epidermolysis bullosa | HST28 | | |
| Afamelanotide for treating erythropoietic protoporphyria | HST27 | | |
| Lumasiran for treating primary hyperoxaluria type 1 | HST25 | | |
| Onasemnogene abeparvovec for treating spinal muscular atrophy | HST15 | | |
| Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency | HST26 | | |
| Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy | HST24 | | |
| Asfotase alfa for treating paediatric-onset hypophosphatasia | HST23 | | |
| Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene | HST22 | | |
| Setmelanotide for treating obesity caused by LEPR or POMC deficiency | HST21 | | |
| Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over | HST20 | | |
| Elosulfase alfa for treating mucopolysaccharidosis type 4A | HST19 | | |
| Atidarsagene autotemcel for treating metachromatic leukodystrophy | HST18 | | |
| Odevixibat for treating progressive familial intrahepatic cholestasis | HST17 | | |
| Givosiran for treating acute hepatic porphyria | HST16 | | |
| Metreleptin for treating lipodystrophy | HST14 | | |
| Volanesorsen for treating familial chylomicronaemia syndrome | HST13 | | |
| Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 | HST12 | | |
| Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations | HST11 | | |
| Patisiran for treating hereditary transthyretin amyloidosis | HST10 | | |
| Inotersen for treating hereditary transthyretin amyloidosis | HST9 | | |
| Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency | HST7 | | |
| Eliglustat for treating type 1 Gaucher disease | HST5 | | |
| Migalastat for treating Fabry disease | HST4 | | |
| Eculizumab for treating atypical haemolytic uraemic syndrome | HST1 | | |